MeiraGTx (NASDAQ:MGTX) has announced that its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1) has been granted Rare Pediatric Disease Designation (RPDD) by the FDA.
According to MeiraGTx, this is the fourth RPDD for inherited renal diseases that the company has received in the past three months, underscoring the transformative therapeutic potential of its proprietary technology platforms.
“Receiving RPDD for an additional program in our ophthalmology pipeline represents another regulatory milestone for the company and demonstrates the groundbreaking therapeutic potential of our technology to address these severe childhood blinding conditions,” Alexandria Forbes, Ph.D., president and CEO of MeiraGTx, commented.
“As we have done with our AIPL1 program, we intend to leverage our manufacturing infrastructure and Specials License along with our clinical expertise in IRDs to work with regulators to expedite the delivery of these potentially life changing treatments to these severely affected children,” Dr. Forbes concluded.
